Ask the Doctor: Is Stem-Cell Transplant an Option?

Dr. Jack Burks

By Dr. Jack Burks
MSAA’s Chief Medical Consultant

Q: In July, MSAA posted an article on their website giving highlights from recent medical meetings. One of the topics covered was stem-cell transplant (SCT), where the treatment involved less-severe preparation for the transplant. In both of the studies mentioned, the individuals who were treated had highly active relapsing MS. Results were positive and study participants experienced improvements in their Expanded Disability Status Scale score.

With these types of results, why isn’t SCT an option for everyone with MS? If I can’t get SCT here in the United States, I have heard that some people are getting SCT in other countries.

A: Stem-cell transplant (SCT) is a complicated subject. In our 2018 edition of MSAA’s MS Research Update, we have a full section devoted to the different types of SCT and how each is performed. It also gives a good overview of recent and upcoming studies with SCT. Anyone interested in learning more may access this publication by going to MSAA’s publications’ page and selecting the 2018 edition of this update.

While SCT is showing some remarkable results for individuals with MS, it is still in the early phases of study and is still considered experimental. SCT is not approved by the United States Food and Drug Administration (FDA) except in clinical trials. Studies so far have been at the Phase I and Phase II levels, and haven’t reached the larger Phase III level – such as those that have been conducted for the approved disease-modifying therapies.

Some may want to know what is taking so long and why they are potentially being deprived of an effective treatment for MS. These individuals need to know that patient safety is the top priority and that the protocol for SCT (referring to the safest procedures and dosages of medications) is still under investigation. Researchers still need to confirm – through clinical trials – the safest and most effective treatment procedures. As with experimental treatments for all conditions, investigators and the FDA must balance safety with effectiveness when evaluating and finalizing treatment protocol.

For FDA approval, the clinical trials must have approved protocols and show a favorable “risk-benefit ratio.” The science is encouraging but more work needs to be completed. Additionally, until the FDA has approved SCT for the treatment of MS, insurance coverage is not available for this procedure.

As you mention in your question, without the approval of SCT for MS in this country, and without the possibility of insurance coverage, some patients are seeking SCT in other countries. This is a very risky endeavor and I can’t warn people enough about the dangers of considering SCT in a country where the medical procedures are not highly regulated. Risks can include treatments that are too severe as well as poor patient monitoring and care, all of which can lead to serious infections, long-term health problems, or worse. If you choose to go to another country for this procedure, make certain it is reputable and not just a commercial venture. Ask your neurologist or an MS center for advice before traveling to receive this procedure.

A safer alternative is to consider treatment with one of the 15 disease-modifying therapies that have been approved by the FDA for the treatment of most forms of MS – with additional possible treatments in the pipeline. The future of MS treatments is very bright. The SCT clinical trials are becoming safer and are encouraging. However, until the clinical trials have been completed and the ideal protocol has been determined, my advice is to work with your neurologist and healthcare team to make sure you are doing everything you can to stay healthy and reduce disease activity while waiting for additional clinical trial results and potential FDA approval of SCT.

Q: I was diagnosed with RRMS in 1994. I have rather significant cognitive fatigue issues from my MS. I began taking the ADHD medication Cylert (pemoline) for this, and it was like night and day for me. The fog burned off and life was now in “real time” again, as opposed to being two or three steps behind on everything. When Cylert came off the market in 2004, I switched to Ritalin® (methylphenidate), and about four years ago, I tried Adderall® (amphetamine and dextroamphetamine) twice per day. I do not know what I would do without this medication. My question is, why don’t we hear about the use of ADHD stimulants for the treatment of this very disabling symptom?

A: I am pleased that Adderall is helping your cognitive fatigue. As noted on MSAA’s website, approximately half of the MS population will experience some type of change in their cognitive abilities during the course of their disease. The most commonly affected cognitive processes in MS are speed-of-information processing, memory, and executive functions (such as organizing, planning, and problem-solving). Up to 80 percent of people with MS also experience the disabling effects of fatigue. With these two symptoms found so frequently, it comes as no surprise that cognitive fatigue might be an issue for someone with MS. Cognitive fatigue can cause an individual to experience reduced attention and slowed-thought processing.

Many MS neurologists prescribe ADHD medications for fatigue, including cognitive fatigue. Provigil® (modafinil) and Nuvigil® (armodafinil) are other options that many find to be helpful. Unfortunately, the problem with prescribing these medications for cognitive fatigue is often a lack of insurance coverage, since they are not approved by the FDA specifically for MS, although exceptions are possible with your neurologist’s persistence. For more information on symptoms and a full listing of medications prescribed, please visit MSAA’s symptoms’ page.

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Jack Burks, MD is the chief medical consultant for MSAA. He is an international MS neurologist, writer, lecturer, and researcher, who assists with the development of new MS therapies as well as new MS centers. He also advises patients, families, MS organizations, and healthcare groups. Dr. Burks is an adjunct professor at Nova Southeastern University and clinical professor of neurology at Florida International University. In addition, he has authored numerous textbooks, chapters, and articles on MS.